Taking polycation gene delivery systems from in vitro to in vivo.

It is widely believed that non-viral gene delivery systems may improve safety and overcome tissue-tropism limitations associated with viral-based gene therapies. Cationic liposome-based gene delivery currently accounts for 9-12% of ongoing gene therapy clinical trials in the United States and Europe. Polycation-based gene delivery is at an earlier development stage. However, both in vitro and in vivo studies have demonstrated that this is an area of much promise. Complexes of polycations with DNA result in major improvements in the control of size, charge, and the hydrophilic-lipophilic characteristics of the transfecting species, when compared with other non-viral systems. This review serves as an introduction to the current status of this field.

[1]  M. Hashida,et al.  Nonviral vectors for in vivo gene delivery: physicochemical and pharmacokinetic considerations. , 1997, Critical reviews in therapeutic drug carrier systems.

[2]  M. Kriegler.  Gene Transfer and Expression: A Laboratory Manual , 1990 .

[3]  H. J. Li,et al.  Interactions between model proteins and deoxyribonucleic acids. , 1976, Biochemistry.

[4]  G. Merlo,et al.  Polyethylenimine-based intravenous delivery of transgenes to mouse lung , 1998, Gene Therapy.

[5]  A. S. Sobolev,et al.  Receptor-mediated endocytosis and nuclear transport of a transfecting DNA construct. , 1992, Experimental cell research.

[6]  V. Alakhov,et al.  Block copolymeric biotransport carriers as versatile vehicles for drug delivery. , 1998, Expert opinion on biological therapy.

[7]  C. Sirlin,et al.  Gene transfer with a series of lipophilic DNA-binding molecules. , 1994, Bioconjugate chemistry.

[8]  Joachim O. Rädler,et al.  Structure of DNA-Cationic Liposome Complexes: DNA Intercalation in Multilamellar Membranes in Distinct Interhelical Packing Regimes , 1997, Science.

[9]  E. Wagner,et al.  Rhinovirus-mediated endosomal release of transfection complexes , 1995, Journal of virology.

[10]  K. Mechtler,et al.  Carbohydrate receptor-mediated gene transfer to human T leukaemic cells. , 1994, Glycobiology.

[11]  I. Wróbel,et al.  Fusion of cationic liposomes with mammalian cells occurs after endocytosis. , 1995, Biochimica et biophysica acta.

[12]  V. Kabanov,et al.  Soluble interpolymeric complexes as a new class of synthetic polyelectrolytes , 1984 .

[13]  R. M. Hays,et al.  Microtubular Disruption Prolongs the Expression of Human Bilirubin-uridinediphosphoglucuronate-glucuronosyltransferase-1 Gene Transferred into Gunn Rat Livers (*) , 1996, The Journal of Biological Chemistry.

[14]  M. Hashida,et al.  Targeted delivery of plasmid DNA to hepatocytes in vivo: optimization of the pharmacokinetics of plasmid DNA/galactosylated poly(L-lysine) complexes by controlling their physicochemical properties. , 1998, The Journal of pharmacology and experimental therapeutics.

[15]  S. V. Vinogradov,et al.  Water-soluble block polycations as carriers for oligonucleotide delivery. , 1995, Bioconjugate chemistry.

[16]  Joseph Zabner,et al.  Cellular and Molecular Barriers to Gene Transfer by a Cationic Lipid (*) , 1995, The Journal of Biological Chemistry.

[17]  P. Cullis,et al.  Nomenclature for synthetic gene delivery systems. , 1997, Human gene therapy.

[18]  R. Kumar,et al.  Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations. , 1994, The Journal of biological chemistry.

[19]  R. Levinsky,et al.  Lipid-mediated enhancement of transfection by a nonviral integrin-targeting vector. , 1998, Human gene therapy.

[20]  R. Diasio,et al.  Absorption, tissue distribution and in vivo stability in rats of a hybrid antisense oligonucleotide following oral administration. , 1995, Biochemical pharmacology.

[21]  A. Kabanov,et al.  Self-assembling complexes for gene delivery : from laboratory to clinical trial , 1998 .

[22]  R. Chung,et al.  Gene therapy for tumors of the central nervous system. , 1998, Surgical oncology clinics of North America.

[23]  Atsushi Harada,et al.  Spontaneous Formation of Polyion Complex Micelles with Narrow Distribution from Antisense Oligonucleotide and Cationic Block Copolymer in Physiological Saline , 1996 .

[24]  E. Wagner.  Effects of membrane-active agents in gene delivery. , 1998, Journal of controlled release : official journal of the Controlled Release Society.

[25]  F. Liu,et al.  Polyethylene glycol-grafted poly-L-lysine as polymeric gene carrier. , 1998, Journal of controlled release : official journal of the Controlled Release Society.

[26]  L. Huang,et al.  Protamine sulfate enhances lipid-mediated gene transfer , 1997, Gene Therapy.

[27]  A. Kabanov,et al.  Self-assembly of polyamine-poly(ethylene glycol) copolymers with phosphorothioate oligonucleotides. , 1998, Bioconjugate chemistry.

[28]  K Mechtler,et al.  The size of DNA/transferrin-PEI complexes is an important factor for gene expression in cultured cells , 1998, Gene Therapy.

[29]  F. Szoka,et al.  The influence of polymer structure on the interactions of cationic polymers with DNA and morphology of the resulting complexes , 1997, Gene Therapy.

[30]  H. Strey,et al.  Improved DNA: liposome complexes for increased systemic delivery and gene expression , 1997, Nature Biotechnology.

[31]  G. Levi,et al.  Size, diffusibility and transfection performance of linear PEI/DNA complexes in the mouse central nervous system , 1998, Gene Therapy.

[32]  F. Szoka,et al.  Polyamidoamine cascade polymers mediate efficient transfection of cells in culture. , 1993, Bioconjugate chemistry.

[33]  C H Wu,et al.  Receptor-mediated in vitro gene transformation by a soluble DNA carrier system. , 1987, The Journal of biological chemistry.

[34]  H. Maeda,et al.  Conjugates of anticancer agents and polymers: advantages of macromolecular therapeutics in vivo. , 1992, Bioconjugate chemistry.

[35]  P. V. Zijl,et al.  Anaerobic bacteria as a gene delivery system that is controlled by the tumor microenvironment , 1997, Gene Therapy.

[36]  M. Hashida,et al.  Physicochemical and pharmacokinetic characteristics of plasmid DNA/cationic liposome complexes. , 1995, Journal of pharmaceutical sciences.

[37]  R. Ghirlando,et al.  Mode of formation and structural features of DNA-cationic liposome complexes used for transfection. , 1993, Biochemistry.

[38]  D. Escande,et al.  Polyethylenimine but Not Cationic Lipids Promotes Transgene Delivery to the Nucleus in Mammalian Cells* , 1998, The Journal of Biological Chemistry.

[39]  A. Kabanov,et al.  DNA complexes with polycations for the delivery of genetic material into cells. , 1995, Bioconjugate chemistry.

[40]  A. Maruyama,et al.  Comb-type polycations effectively stabilize DNA triplex. , 1997, Bioconjugate chemistry.

[41]  A. Rolland.  From genes to gene medicines: recent advances in nonviral gene delivery. , 1998, Critical reviews in therapeutic drug carrier systems.

[42]  M. Monsigny,et al.  Specific gene transfer mediated by lactosylated poly-L-lysine into hepatoma cells. , 1993, Nucleic acids research.

[43]  H. D. Liggitt,et al.  Factors influencing the efficiency of cationic liposome-mediated intravenous gene delivery , 1997, Nature Biotechnology.

[44]  C. Wu,et al.  Receptor-mediated gene delivery and expression in vivo. , 1988, The Journal of biological chemistry.

[45]  Leaf Huang,et al.  Potentiation of cationic liposome-mediated gene delivery by polycations. , 1996, Biochemistry.

[46]  P. De Camilli,et al.  Amphiphysin I Antisense Oligonucleotides Inhibit Neurite Outgrowth in Cultured Hippocampal Neurons , 1998, The Journal of Neuroscience.

[47]  K. Ulbrich,et al.  Characterization of vectors for gene therapy formed by self-assembly of DNA with synthetic block co-polymers. , 1996, Human gene therapy.

[48]  Leaf Huang,et al.  Lipidic vector systems for gene transfer. , 1997, Critical reviews in therapeutic drug carrier systems.

[49]  J. C. Perales,et al.  An evaluation of receptor-mediated gene transfer using synthetic DNA-ligand complexes. , 1994, European journal of biochemistry.

[50]  J. Guy,et al.  Delivery of DNA into mammalian cells by receptor-mediated endocytosis and gene therapy , 1995, Molecular biotechnology.

[51]  A. Kabanov,et al.  Reduction of fibronectin expression by intravitreal administration of antisense oligonucleotides , 1999, Nature Biotechnology.

[52]  A. Kabanov,et al.  Efficient transformation of mammalian cells using DNA interpolyelectrolyte complexes with carbon chain polycations. , 1993, Bioconjugate chemistry.